Ganaxolone (3α-hydroxy-3β-methyl-5α-pregnan-20-one, (3α,5α)-3-hydroxy-3-methylpregnan-20-one, or CCD 1042) is a synthetic neurosteroid analogue that acts as a modulator of GABAA receptors. Ganaxolone has been tested for safety in clinical trials, and has relatively modest side effects even at very high doses. It has shown promise for treating temporal lobe seizures as well as catamenial epilepsy. Temporal lobe epilepsy is characterized by recurrent seizures that originate in the temporal lobe of the brain. Temporal lobe seizures can involve sensory changes as well as memory disturbances. Temporal lobe seizures are characterized as partial seizures, including simple and complex, and generalized seizures such as secondarily generalized tonic-clonic seizures. Catamenial epilepsy occurs in women with preexisting epilepsy and is characterized by seizure exacerbations during the menstrual cycle, such as in the premenstrual period. Ganaxolone is also under study for the treatment of posttraumatic stress disorder and Fragile-X syndrome. Ganaxolone has been approved by the U.S. FDA for the treatment of protocadherin 19 gene (PCDH19) female epilepsy.
Because ganaxolone is well-tolerated in adults and children, it is desirable to uncover additional uses for ganaxolone.